Klinische Monatsblätter für Augenheilkunde, Table of Contents Klin Monbl Augenheilkd 2025; 242(03): 189-190DOI: 10.1055/a-2511-6878 Editorial Pharmakotherapie von hereditären Netzhautdystrophien Claudia Priglinger , Katarina Stingl Recommend Article Abstract Buy Article Full Text References Literatur 1 Schneider N, Sundaresan Y, Gopalakrishnan P. et al. Inherited retinal diseases: Linking genes, disease-causing variants, and relevant therapeutic modalities. Prog Retin Eye Res 2022; 89: 101029 2 Cremers FPM, Lee W, Collin RWJ. et al. Clinical spectrum, genetic complexity and therapeutic approaches for retinal disease caused by ABCA4 mutations. Prog Retin Eye Res 2020; 79: 100861 3 Cideciyan AV, Aleman TS, Swider M. et al. Mutations in ABCA4 result in accumulation of lipofuscin before slowing of the retinoid cycle: a reappraisal of the human disease sequence. Hum Mol Genet 2004; 13: 525-534 4 Campochiaro PA, Iftikhar M, Hafiz G. et al. Oral N-acetylcysteine improves cone function in retinitis pigmentosa patients in phase I trial. J Clin Invest 2020; 130: 1527-1541 5 Schulz A, Specchio N, de Los Reyes E. et al. Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study. Lancet Neurol 2024; 23: 60-70